Oligo Insights | Overview of Domestic and Global Oligonucleotide Industry Developments – Q1 2025
CSPC
Innovative Drug Research and Development, Manufacturer
Arnatar
Antisense Technology Drug Developer
Hepa Thera
Innovative Drug Developer in the Field of Hepatology
Sirius Therapeutics
Oligonucleotide Drug Developer
Innovation in China
01 CSPC
CSPC's Second siRNA Drug Approved for Clinical Trials! Potential for Twice-Yearly Dosing in Hypertension Treatment
On December 23, 2024, CSPC announced that its self-developed Class 1 new chemical drug, SYH2062 Injection (a double-stranded small interfering RNA (siRNA) candidate drug), has been approved by the National Medical Products Administration of the People's Republic of China to conduct clinical trials in China for the treatment of hypertension.
SYH2062 is a siRNA candidate drug delivered by conjugated N-acetylgalactosamine (GalNAc) that can target and inhibit angiotensinogen (AGT), under development for the treatment of hypertension.
SYH2062 achieves a more durable gene silencing effect through optimized sequence and chemical modification strategies, with the potential to become a stable blood pressure control drug administered once every six months. Preclinical studies show that the duration of SYH2062's pharmacological activity is significantly longer than similar siRNA products, with expected advantages of prolonged efficacy, good safety, and high patient compliance.
02 Arnatar
Arnatar's siRNA Drug ART101 Injection Approved for Clinical Use to Treat Hypertension
December 30, 2024Arnatar's self-developed new chemical entity Class I new drug ART101 injection (siRNA drug) has received clinical trial implied permission from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) (Acceptance No.: CXHL2401113). The drug is intended for the treatment of hypertension.
Disclosed preclinical and clinical studies have shown that oligonucleotide drugs targeting AGT can reduce the production of hepatic AGT by inhibiting liver AGT mRNA levels, thereby decreasing systemic AGT levels and achieving the purpose of lowering blood pressure. ART101 Injection, as a siRNA drug independently developed by Arnatar, achieves its antihypertensive effect by targeting and inhibiting the expression of hepatic AGT mRNA, reducing the generation of AGT protein.
03 Hepa Thera
Hepa Thera HT-101 and HT-102 Combination Therapy Completes First Patient Dosing in Phase II Clinical Trial
On December 30, 2024, Hepa Thera successfully completed the first dosing of subjects in the Phase II combination study of HT-101 (GalNAc-siRNA) injection and HT-102 (humanized monoclonal antibody) injection. This study aims to fully explore the efficacy and safety under longer dosing intervals, striving to bring better treatment options to patients.
Previous phase data showed that subjects in the high-dose group of HT-101 achieved HBsAg clearance after only two administrations, the best among similar products globally; for HT-102, HBsAg rapidly decreased by more than 2 logs within two days post-administration. Both drugs demonstrated good safety profiles.
04 Sirius Therapeutics
Sirius Therapeutics' Another siRNA Therapy Clinical Trial Application Accepted by China's NMPA
December 31, 2024, Shanghai, China, and San Diego, USA: Sirius Therapeutics (SHANGHAI) Co., Ltd. ("Sirius Therapeutics"), announced today that its Investigational New Drug (IND) application for SRSD216 has been successfully accepted by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) in China.
SRSD216 is a next-generation siRNA therapy for the treatment of hyperlipoproteinemia (a).It specifically modulates the LPA gene, reducing hepatic Apo(a) production and lowering circulating Lp(a) levels. Preclinical in vivo studies have shown that a single dose can reduce Lp(a) levels by nearly 100%, with effects lasting over six months, and no significant safety events were observed.
Overseas Hotspots
01 Ionis
TRYNGOLZA™ (olezarsen) Successfully Receives FDA Approval, Adding a New Member to Approved ASO Therapies
FCS is a rare genetic severe hypertriglyceridemia (sHTG) that prevents the body from breaking down fats and severely impairs the body's ability to remove triglycerides from the blood due to impaired lipoprotein lipase (LPL) function.TRYNGOLZA is the first FDA-approved treatment that significantly and substantially reduces triglyceride levels in adult patients with FCS., and with the cooperation of an appropriate diet (≤20 grams of fat per day), provides a clinically significant reduction in AP events.
02 Alloy
The First ASO Therapy Strategic Collaboration in 2025: Alloy Could Receive Over $420 Million in Milestone Payments
On January 7, 2025, ASO design company Alloy Therapeutics announced a target-specific collaboration and licensing agreement with Sanofi for the use of its novel and proprietary AntiClastic antisense platform on central nervous system (CNS) targets.
In return,Sanofi will provide Alloy with upfront license fees and near-term preclinical milestone payments of up to $27.5 million. Alloy is also eligible to receive over $400 million in discovery, development, and commercial milestone payments, as well as tiered royalties on product sales generated from any collaboration.。
03 Alnylam
Alnylam Announces Full-Year 2024 Results and Provides Guidance for Over $2 Billion in 2025, Igniting New Momentum in the Small Nucleic Acid Industry
Cambridge, MA, January 12, 2025 – Alnylam announced preliminary global net product revenues for ONPATTRO, AMVUTTRA, GIVLAARI, and OXLUMO for the fourth quarter and full year of 2024.$1.646 billion, a year-on-year increase of 33%。
2025 will be a pivotal turning point for Alnylam's TTR franchise, with the launch of vutrisiran in ATTR-CM, and its net product sales guidance reflects potential significant growth.Guidance for Comprehensive Net Product Revenue in 2025: $2.05 Billion to $2.25 Billion, making the company hopeful to achieve Alnylam P5x25 non-GAAP profitability target.
PATIENTs: Over 500,000 patients globally use Alnylam RNAi therapeutic drugs PRODUCTS: Launch more than 6 marketed products in the fields of rare diseases and common diseases. PIPELINE: Over 20 clinical projects; more than 10 in late stages; over 4 new INDs (Investigational New Drug applications) added annually. PERFORMANCEPerformance: By the end of 2025, the compound annual growth rate of revenue reached 240%. PROFITABILITYProfitability: Achieving sustainable non-GAAP profitability within the specified timeframe
About Ouli Biotech
Suzhou Oli Biopharmaceuticals Technology Co., Ltd. is a professional medicinal nucleic acid CRDMO.The company provides customers with "one-stop" services including nucleic acid drug discovery, laboratory R&D, process and analytical development, CMC services, API production, and drug registration. The technical team of Oulibio comes from one of the earliest teams in China to engage in the development of nucleic acid drug production processes and CMC research, possessing extensive experience in project development and product registration. Currently, the company has established four leading core technology platforms in the industry: solid-phase synthesis of nucleic acids, chemical modification and conjugation, process development and analysis, as well as CMC pharmaceutical research. It has also entered into in-depth collaborations with multiple pharmaceutical companies and biotechnology firms both domestically and internationally.
In 2023, the company's 3,000 square meter GMP-standard pilot platform officially began offering services to help clients address critical challenges such as small nucleic acid drug process scale-up and CMC pharmaceutical research. The company will continue to provide compliant, high-quality, reliable, and efficient services to enhance clients' R&D efficiency, expedite product registration and market entry, and collectively support the entire process of small nucleic acid drugs from preclinical to commercial production.
Contact Us
Email: order@olipharma.com
Phone: 15336788818
Official Website: www.olipharma.com
R&D Center: No. 168, Yuanfeng Road, Yushan Town, Kunshan City, Jiangsu Province
Production Base: Building C3, Phase I Factory, No. 999 Yishanhu Road, Guoxiang, Wuzhong District, Suzhou City, Jiangsu Province
Common Technology Platform: 202, 2nd Floor, Building 2, No. 21 Baoshen South Street, Beijing Daxing District Biomedical Industry Base